Eliglustat +/− Imiglucerase for Gaucher Disease

(ELIKIDS Trial)

Recruiting at 65 trial locations

Overseen By Trial Transparency email recommended (Toll free number for US & Canada)

Age: < 18

Sex: Any

Trial Phase: Phase 3

Sponsor: Sanofi

Must be taking: Enzyme replacement therapy

Must not be taking: Substrate reduction therapy

Disqualifiers: Splenectomy, Transfusion dependent, Cardiac disease, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

57 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

Primary Objective: Evaluate the safety and pharmacokinetics of eliglustat in pediatric patients (≥2 to \<18 years old). Secondary Objective: Evaluate the efficacy of eliglustat and quality of life in pediatric patients (≥2 to \<18 years old).

Will I have to stop taking my current medications?

The trial does not specify if you need to stop taking your current medications. However, you must have been on enzyme replacement therapy for a certain period before joining, and you cannot have used substrate reduction therapy in the last 6 months.

Is Eliglustat safe for humans?

Eliglustat has been shown to be generally safe for humans, with most side effects being mild or moderate and unrelated to the treatment. Long-term studies have not identified any new or serious safety concerns, and it was well-tolerated over several years of use in patients with Gaucher disease type 1. ^{1 2 3 4 5}

How does the drug Eliglustat differ from other treatments for Gaucher Disease?

Eliglustat is unique because it is an oral medication, unlike the traditional enzyme replacement therapies like imiglucerase, which are given through intravenous infusions. It works by reducing the production of certain fats that accumulate in Gaucher disease, offering a convenient alternative for patients who are compatible with its metabolism. ^{1 3 5 6 7}

What data supports the effectiveness of the drug Eliglustat for Gaucher Disease?

Research shows that Eliglustat, an oral drug, is effective in maintaining stability in patients with Gaucher Disease type 1, similar to the standard enzyme therapy, imiglucerase. It has been shown to decrease spleen and liver size and improve blood parameters like hemoglobin and platelet counts in both new and previously treated patients. ^{1 2 3 5 8}

Who Is on the Research Team?

Clinical Sciences & Operations

Principal Investigator

Sanofi

Are You a Good Fit for This Trial?

This trial is for kids aged 2-17 with Gaucher Disease types 1 or 3. They must have been on enzyme replacement therapy (ERT) and meet certain health criteria. Girls post-puberty need a negative pregnancy test and agree to contraception or abstinence.

Inclusion Criteria

I am between 2 and 17 years old.

I've been on enzyme replacement therapy for at least 24 months and meet specific treatment goals.

I've been on enzyme replacement therapy for over 36 months and have severe Gaucher's disease symptoms.

Exclusion Criteria

I have been on substrate reduction therapy for Gaucher's disease within the last 6 months.

I have health issues like heart, liver problems, or other serious conditions.

I cannot or do not want to take imiglucerase due to allergies or personal choice.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

8 weeks

Primary Analysis Treatment

Participants receive eliglustat with or without imiglucerase to evaluate safety and pharmacokinetics

52 weeks

Regular visits for monitoring and assessment

Long-term Treatment

Continued treatment with eliglustat or combination therapy based on clinical response

52 weeks

Extension

Participants continue treatment up to Week 364 if clinical benefit is demonstrated

Up to 260 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Eliglustat GZ385660
• Imiglucerase GZ437843

Trial Overview The study tests the safety and effects of Eliglustat alone, or combined with Imiglucerase in children with Gaucher Disease. It looks at how their bodies handle the drugs (pharmacokinetics), changes in their condition, and life quality.

How Is the Trial Designed?

Group I: Cohort 2: Eliglustat plus imiglucerase Experimental Treatment 2 Interventions

Eliglustat plus imiglucerase for three years, at the dose of enzyme replacement therapy received before enrollment. After Week 52, Cohort 2 patients will switch to eliglustat monotherapy for the remainder of the study if the desired clinical response has been achieved.

Group II: Cohort 1: Eliglustat monotherapy Experimental Treatment 1 Intervention

Eliglustat for at least two years. Cohort 1 patients that experience significant clinical decline will receive rescue treatment. Rescue Treatment Step 1: Switch from eliglustat to imiglucerase monotherapy. Rescue Treatment Step 2: Patients who after 6 months of rescue therapy with imiglucerase monotherapy do not show improvement in the parameter(s) that led to the switch from eliglustat to imiglucerase, will then receive combination therapy with eliglustat + imiglucerase.

Who Is Running the Clinical Trial?

Sanofi

Lead Sponsor

Trials

2,246

Recruited

4,085,000+

Paul Hudson

Sanofi

Chief Executive Officer since 2019

Degree in Economics from Manchester Metropolitan University

Christopher Corsico

Sanofi

Chief Medical Officer

MD from Cornell University, MPH in Chronic Disease Epidemiology from Yale University

Citations

1. United States pubmed.ncbi.nlm.nih.gov

Stability is maintained in adults with Gaucher disease type 1 switched from velaglucerase alfa to eliglustat or imiglucerase: A sub-analysis of the eliglustat ENCORE trial. [2020]

2. United States pubmed.ncbi.nlm.nih.gov

Clinical response to eliglustat in treatment-naïve patients with Gaucher disease type 1: Post-hoc comparison to imiglucerase-treated patients enrolled in the International Collaborative Gaucher Group Gaucher Registry. [2020]

3. Netherlands pubmed.ncbi.nlm.nih.gov

Management and monitoring recommendations for the use of eliglustat in adults with type 1 Gaucher disease in Europe. [2022]

4. United States pubmed.ncbi.nlm.nih.gov

Outcomes after 8 years of eliglustat therapy for Gaucher disease type 1: Final results from the Phase 2 trial. [2021]

5. Austria pubmed.ncbi.nlm.nih.gov

Long-term eliglustat treatment of Gaucher patients over up to 10 years in Vienna. [2022]

6. United States pubmed.ncbi.nlm.nih.gov

Eliglustat maintains long-term clinical stability in patients with Gaucher disease type 1 stabilized on enzyme therapy. [2022]

7. Germany pubmed.ncbi.nlm.nih.gov

Skeletal improvement in patients with Gaucher disease type 1: a phase 2 trial of oral eliglustat. [2021]

8. New Zealand pubmed.ncbi.nlm.nih.gov

Eliglustat: A Review in Gaucher Disease Type 1. [2018]